CellAct Pharma, a developer of innovative treatments for cancer,
announced today that the Committee for Orphan Medicinal Products of the
European Medicines Agency (EMA (News - Alert)) has granted orphan drug designation to
the company's CAP7.1, an adapted version of the well-established
anticancer agent etoposide, for the treatment of biliary tract cancers.
European Union (EU) orphan drug designation is given to products for the
diagnosis, prevention or treatment of rare diseases that are
life-threatening or very serious. A disease is defined as rare in the EU
if it affects fewer than five in 10,000 people. Biliary tract cancer
affects approximately 1.7 in 10,000 people in the EU. This is equivalent
to a total of around 87,000 people.

The granting of EU orphan drug designation provides CellAct with
development and commercial incentives, including a 10-year period of
market exclusivity, access to a centralized review process, protocol
assistance and scientific advice during product development, waiving or
reduction of certain fees, and eligibility for grants and R&D support
initiatives. CellAct is currently recruiting patients for a randomized,
multicenter, proof-of-concept phase 2 study with CAP7.1 in adults with
refractory biliary trac carcinomas in Germany (www.cap7-1.com).

"Following diagnosis, there are very few treatments options for biliary
tract cancer patients. Granting of orphan drug designation could help
expedite the regulatory process, if our clinical studies prove to be
successful, and potentially get CAP7.1 to patients sooner," stated Nalân
Utku, M.D., Chief Executive Officer of CellAct. "Recruitment into our
phase 2 clinical study in Germany is progressing on schedule, with
additional centers coming online each month."

A previous clinical study in adults with multiple and heavily pretreated
solid tumors, including biliary tract cancers, showed promising safety
and tolerability following treatment with CAP7.1. Many of these patients
also showed signs of efficacy.

In addition to biliary tract cancers, a phase II study is also
recruiting patients with therapy-refractory non-small and small cell
lung cancers.

CellAct Pharma is focused on the development of innovative therapeutics
for the treatment of cancer. CellAct's drug candidates target and
modulate human molecules that have specific functions in tumor growth. A
first-in-class small molecule compound, CAP7.1 is currently enrolling
patients in randomized, multicenter clinical Phase II studies for the
treatment of biliary tract cancers, Non-small cell lung cancers and
small cell lung cancers. In addition to venture capital funding, CellAct
has received a €0.7 million grant from the German ministry for education
and science (Bmbf) to support this program. For further information
visit www.cellact.eu.